(19)
(11) EP 4 249 472 A3

(12) EUROPEAN PATENT APPLICATION

(88) Date of publication A3:
13.12.2023 Bulletin 2023/50

(43) Date of publication A2:
27.09.2023 Bulletin 2023/39

(21) Application number: 23173467.4

(22) Date of filing: 27.05.2016
(51) International Patent Classification (IPC): 
A01N 43/90(2006.01)
C07D 239/70(2006.01)
A61K 31/519(2006.01)
(52) Cooperative Patent Classification (CPC):
A61K 31/519; A61K 31/711; C07D 239/70; Y02A 50/30; C12N 15/63
(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

(30) Priority: 30.05.2015 US 201562168726 P

(62) Application number of the earlier application in accordance with Art. 76 EPC:
16804178.8 / 3310169

(71) Applicant: PTC Therapeutics, Inc.
South Plainfield, NJ 07080 (US)

(72) Inventors:
  • NARYSHKIN, Nikolai, A.
    East Brunswick, NJ 08816 (US)
  • DAKKA, Amal
    Whitehouse Station, NJ 08889 (US)

(74) Representative: Jones Day 
Rechtsanwälte, Attorneys-at-Law, Patentanwälte Prinzregentenstrasse 11
80538 München
80538 München (DE)

   


(54) METHODS FOR MODULATING RNA SPLICING


(57) In one aspect, described herein is a recognition element for splicing (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene construct comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.





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